TY  - JOUR
AU  - Martinez Dominguez, Maria Victoria
AU  - Falke, Lisa
AU  - Beretta, Stefania
AU  - Dauer, Katharina
AU  - Wagner, Karl G.
AU  - Boeckers, Tobias M.
TI  - Manufacturing and testing of a polymer formulation for long-time release delivery of antisense oligonucleotides in hiPSC-derived motoneurons
JO  - Journal of drug delivery science and technology
VL  - 119
SN  - 1773-2247
CY  - Paris
PB  - Ed. de Santé
M1  - DZNE-2026-00294
SP  - 108171
PY  - 2026
AB  - Antisense oligonucleotides (ASOs) are an emerging therapeutic approach for genetically induced neurological disorders such as spinal muscular atrophy (SMA). For SMA, the Food and Drug administration (FDA)-approved ASO Nusinersen (Spinraza®) restores exon 7 inclusion in survival motor neuron (SMN) 2, leading to improved motoneuron survival. However, current administration to patients requires repeated intrathecal (IT) injections via lumbar puncture, an invasive procedure with considerable side effects. There is therefore an urgent need for more efficient and sustained drug-delivery strategies capable of overcoming these limitations. In this study, we developed and tested a system for the controlled, long-term release of Nusinersen ASO. Using motoneurons derived from human induced pluripotent stem cells (hiPSCs) from SMA and control patients, two formulations were evaluated: (I) pure ethylene vinyl acetate (EVA, 40
LB  - PUB:(DE-HGF)16
DO  - DOI:10.1016/j.jddst.2026.108171
UR  - https://pub.dzne.de/record/285737
ER  -